Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …
technologies has opened up the possibility of directly targeting and modifying genomic …
Pathophysiology of sickle cell disease
Since the discovery of sickle cell disease (SCD) in 1910, enormous strides have been made
in the elucidation of the pathogenesis of its protean complications, which has inspired recent …
in the elucidation of the pathogenesis of its protean complications, which has inspired recent …
[HTML][HTML] Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000–2021: a systematic analysis from the Global Burden of Disease …
Background Previous global analyses, with known underdiagnosis and single cause per
death attribution systems, provide only a small insight into the suspected high population …
death attribution systems, provide only a small insight into the suspected high population …
Blood rheology: key parameters, impact on blood flow, role in sickle cell disease and effects of exercise
E Nader, S Skinner, M Romana, R Fort… - Frontiers in …, 2019 - frontiersin.org
Blood viscosity is an important determinant of local flow characteristics, which exhibits shear
thinning behavior: it decreases exponentially with increasing shear rates. Both hematocrit …
thinning behavior: it decreases exponentially with increasing shear rates. Both hematocrit …
Continuous evolution of SpCas9 variants compatible with non-G PAMs
The targeting scope of Streptococcus pyogenes Cas9 (SpCas9) and its engineered variants
is largely restricted to protospacer-adjacent motif (PAM) sequences containing G bases …
is largely restricted to protospacer-adjacent motif (PAM) sequences containing G bases …
[HTML][HTML] A red carpet for iron metabolism
200 billion red blood cells (RBCs) are produced every day, requiring more than 2× 10 15
iron atoms every second to maintain adequate erythropoiesis. These numbers translate into …
iron atoms every second to maintain adequate erythropoiesis. These numbers translate into …
[HTML][HTML] Crizanlizumab for the prevention of pain crises in sickle cell disease
KI Ataga, A Kutlar, J Kanter, D Liles… - … England Journal of …, 2017 - Mass Medical Soc
Background The up-regulation of P-selectin in endothelial cells and platelets contributes to
the cell–cell interactions that are involved in the pathogenesis of vaso-occlusion and sickle …
the cell–cell interactions that are involved in the pathogenesis of vaso-occlusion and sickle …
Understanding sickle cell disease: causes, symptoms, and treatment options
C Elendu, DC Amaechi, CE Alakwe-Ojimba… - Medicine, 2023 - journals.lww.com
Sickle cell disease (SCD) is a hereditary blood disorder characterized by the production of
abnormal hemoglobin molecules that cause red blood cells to take on a crescent or sickle …
abnormal hemoglobin molecules that cause red blood cells to take on a crescent or sickle …
Global burden of sickle cell anaemia in children under five, 2010–2050: modelling based on demographics, excess mortality, and interventions
Background The global burden of sickle cell anaemia (SCA) is set to rise as a consequence
of improved survival in high-prevalence low-and middle-income countries and population …
of improved survival in high-prevalence low-and middle-income countries and population …
[HTML][HTML] Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates
Background Reliable estimates of populations affected by diseases are necessary to guide
efficient allocation of public health resources. Sickle haemoglobin (HbS) is the most …
efficient allocation of public health resources. Sickle haemoglobin (HbS) is the most …